The CF Patient Registry is an observational study. It does not involve medical treatments and serves only as an epidemiologic record of demographics, characteristics of disease and treatment, utilization patterns, and clinical outcomes.

Lamprene®/Clofazimine, is a product of the pharmaceutical company named Novartis Pharmaceuticals Corporation. Lamprene®/Clofazimine is approved by FDA (the U.S Food and Drug Administration) for the treatment of leprosy. It is being tested in non-Novartis clinical studies for drug resistant tuberculosis and non-tuberculous mycobacteria (NTM).

If you have been diagnosed with NTM, then your doctor may decide that this infection can be treated with Lamprene®/Clofazimine. This medicine is provided to you in an expanded access program. This means that this medicine is not registered for the treatment of NTM, but it can be used in special situations where there are no other possible treatments. For example, this may be because you have a type of Mycobacterial infection that is resistant or failed to respond optimally to other drugs, or because you have had side effects that prevent the use of other drugs.

The goal of this cohort and biorepository is to collect data and blood specimens on individuals with Nontuberculous Mycobacteria (NTM), pulmonary disease, and healthy adults to better understand the illness and ultimately improve the care and survival of those with these conditions.

The purpose of this study is to better understand treatment practices for Mycobacterium avium complex (MAC) disease. This study is comparing the effectiveness and tolerability of taking 2 drugs compared to 3 drugs in people with MAC disease.

The purpose of this study is to evaluate the effect and safety of amikacin liposome inhalation suspension (ALIS) study treatment on patient-reported symptoms in subjects newly diagnosed with NTM lung infection caused by MAC who have not started standard treatment. Around 250 subjects 18 years and older with newly diagnosed NTM lung infection caused by MAC who have not started standard treatment are expected to participate in this study. The study will last around 17.5 months from the Screening visit to the end of the study.

This is a prospective, multi-center, observational study in pregnant women with cystic fibrosis (CF) to characterize forced expiratory volume in 1 second (FEV1) changes based on exposure to highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators. The key factors contributing to the change in lung function during pregnancy and for 2 years post-delivery will be evaluated along with assessment of fetal and maternal outcomes.
The total duration of participation for each participant is expected to be about 3 years (up to 35 months). Women will be enrolled in the first trimester of pregnancy and assessed every 3 months during pregnancy and during the first year after delivery, then every 6 months for an additional year. Over the course of the study, CF clinical data, patient-reported outcomes, questionnaires, obstetrical outcomes, infant growth, child development outcomes, baseline CF-related therapies and co-morbidities will be collected to enable evaluation of changes from before pregnancy to during pregnancy and post-delivery.
MAYFLOWERS participants will be provided an opportunity to participate in an optional continuous glucose monitoring (CGM) sub-study to evaluate glucose control in pregnancy. Participants will undergo CGM sensor placement and data collection as part of the MAYFLOWERS study.

The purpose of this study is to learn more about the safety and effectiveness of the drug omadacyline for people with Nontuberculous Mycobacterial (NTM) pulmonary disease, caused by a type of mycobacteria called Mycobacterium abscessus complex (MABc). Participants will be randomly assigned to receive either omadacycline or placebo, with a 60% chance of being treated with omadacycline and a 40% chance of being treated with placebo. About 75 men and women with NTM lung infection caused by MABc, ages 18 and up, will be in this study, with an estimated 4 participants being enrolled from the MUSC site. Participants will be in this study for approximately 5 months in total, beginning with the Screening visit and continuing until their last study visit.

Nontuberculous mycobacteria (NTM) cause a chronic pulmonary infection associated with cough, fatigue, and shortness of breath. We seek to develop and test a disease-specific patient-reported outcome measure (PROM) in well-characterized populations of patients with NTM pulmonary disease. Ultimately, a validated PROM for individuals with NTM pulmonary disease will fill a critical need for effective NTM treatments by improving clinical trial design and identifying a meaningful endpoint that can guide therapeutic development.

This is a pivotal Phase 2/3, double-blind, placebo-controlled study of oral study drug epetraborole + OBR (Optimized background regimen) versus placebo + OBR in patients with treatment-refractory MAC lung disease. A total of approximately 314 patients will be enrolled in this Phase 2/3 study. For the Phase 2 and Phase 3 parts of the study, the duration of study participation for each patient, not including
Screening, will be up to approximately 19 months. During the conduct of the study, an independent Data and Safety Monitoring Board (DSMB) will be responsible for periodic review of unblinded study safety data by performing a qualitative and quantitative safety assessment.

The Program will allow access to brensocatib for patients who have completed the INS1007-301 ASPEN Clinical Trial. Patients will receive brensocatib 10 mg orally once daily. Eligible, compliant patients may receive brensocatib in this program until the drug is commercially available or until Insmed terminates the program.